Clinical experience with Raxone (idebenone) in the treatment of patients with Leber s Hereditary Optic Neuropathy (LHON)

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1 Clinical experience with Raxone (idebenone) in the treatment of patients with Leber s Hereditary Optic Neuropathy (LHON) Günther Metz, Santhera Pharmaceuticals

2 Disclaimer 2 This presentation is not and under no circumstances to be construed as a solicitation, offer, or recommendation, to buy or sell securities issued by Santhera Pharmaceuticals Holding AG. Santhera Pharmaceuticals Holding AG makes no representation (either express or implied) that the information and opinions expressed in this presentation are accurate, complete or up to date. Santhera Pharmaceuticals Holding AG disclaims, without limitation, all liability for any loss or damage of any kind, including any direct, indirect or consequential damages, which might be incurred in connection with the information contained in this presentation. This presentation expressly or implicitly contains certain forward-looking statements concerning Santhera Pharmaceuticals Holding AG and its business. Certain of these forward-looking statements can be identified by the use of forward-looking terminology or by discussions of strategy, plans or intentions. Such statements involve certain known and unknown risks, uncertainties and other factors, which could cause the actual results, financial condition, performance or achievements of Santhera Pharmaceuticals Holding AG to be materially different from any expected results, performance or achievements expressed or implied by such forward-looking statements. There can be no guarantee that any of the research and/or development projects described will succeed or that any new products or indications will be brought to market. Similarly, there can be no guarantee that Santhera Pharmaceuticals Holding AG or any future product or indication will achieve any particular level of revenue. In particular, management s expectations could be affected by, among other things, uncertainties involved in the development of new pharmaceutical products, including unexpected preclinical and clinical trial results; unexpected regulatory actions or delays or government regulation generally; the Company s ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry, and general public pricing and other political pressures. Santhera Pharmaceuticals Holding AG is providing the information in this new release as of the date of the publication, and does not undertake any obligation to update any forward-looking statements contained herein as a result of new information, future events or otherwise.

3 Leber s Hereditary Optic Neuropathy 3 Rare mitochondrial genetic disease Predominantly young males affected Rapid and mostly permanent loss of central vision by functional loss and degeneration of optic nerve cells No treatment available In an (ultra-) orphan disease, unique challenges to provide sufficient and robust data in clinical development for regulatory filing need to be overcome.

4 Therapeutic objectives 4 Stabilization of good residual vision Promotion of clinically relevant recovery in a window of opportunity for treatment of up to several years when retinal ganglion cells are still viable VA NATURAL HISTORY Off - chart RECOVERY ONSET 0 STABILIZATION YEARS

5 Idebenone (Raxone ) 5 synthetic short-chain benzoquinone unique physicochemical properties and distinct mode of action (MoA) MoA substrate of the cytosolic enzyme NQO1 capable to transport electrons to the inner mitochondrial membrane effective in restoring cellular energy levels acts as an antioxidant in the mitochondria

6 Expanded Access Program (EAP) 6 The EAP was set up by Santhera in Nov to respond to unsolicited requests from physicians to get access to idebenone. The focus is on patients in urgent medical need with recent onset of symptoms (<12 months). Drug is provided under country specific regulations for Named Patient Programs (EU,RoW) or emergency INDs (USA). Patients were typically treated with idebenone at 900 mg/d and followed-up in 3 monthly intervals. Safety and visual acuity data (ETDRS) were collected using standardized report forms.

7 EAP LHON Database 7 The EAP represents the largest collection of LHON patients treated with idebenone so far Visual acuity data are available from 93 patients treated for up to 36 months. 36 sites participated worldwide.

8 EAP: Patient Demographics 8 Data are presented for 69 patients carrying one of the 3 major mutations and having a post baseline assessment. The demographics are in line with the expected natural history.

9 Visual Acuity Outcome Analyses 9 Clinically Relevant Recovery (CRR) From off-chart to reading 5 letters (1 line) or Improvement of 10 letters (2 lines) on chart Stabilization Patients who do not progress to logmar 1.0 (20/200)

10 Prevention of further VA loss in 57% of patients with vision below logmar Analysis in the subset of patients with best VA below logmar 1.0 at start of treatment (n=21): Mean time since onset at start of treatment: 4.6 m Mean follow-up time: 15.5 m

11 Clinically relevant recovery (CRR) in a large proportion of patients (49%) 11 Recovery is observed in patients carrying any of major 3 mutations and the rate is largest for carriers of the T14484C mutation. Analyses from the entire EAP efficacy population (n=69):

12 Treatment duration at first recovery for patients with CRR (n=34) 12 The majority of patients with CRR have experienced their first recovery after 6 months of treatment. Additional patients show recovery after longer therapy. First recovery after more than 12 months of treatment is rare.

13 The magnitude of VA recovery increases with treatment duration 13 The mean recovery at the last observation was > 9 lines (46 letters). Analyses for eyes with clinically relevant recovery observed after 6 months of treatment and where 12 month follow-up data were available (n=15):

14 Summary 14 The EAP represents a valuable source of information on clinical, real world experience in the treatment of patients with LHON with Raxone (idebenone). A large proportion of patients experienced either recovery of lost VA and / or prevention of further VA loss in eyes with residual good VA. The use of Raxone (idebenone) was safe and well tolerated. Data from an Expanded Access Program can be included in a regulatory dossier for a marketing authorization application.

15 Thank you 15 Santhera likes to acknowledge the treating physicians and participating patients. The presentation will be available on the Santhera webpage. Raxone (idebenone) has a temporary marketing authorization in France (ATU de cohorte) and is under review in a centralized procedure by the European Medicines Agency (EMA).